Primary results from phase II ZUMA-2 trial of KTE X19 to treat relapsed or refractory mantle cell lymphoma. Kite/Gilead

Kite, a Gilead Company announced primary results from ZUMA-2 , a global, multicenter, single-arm, open-label Phase II study of KTE X19, an investigational CD19 chimeric antigen receptor (CAR) T cell therapy, in adult patients with relapsed or refractory mantle cell lymphoma (MCL). After a single infusion of KTE X19, the best objective response via independent radiologic central review (n=60 evaluable for efficacy analysis) was 93 percent, with 67 percent of patients having achieved a complete response. These findings were presented at the 61st American Society of Hematology (ASH) Annual Meeting & Exposition, in Orlando from December 7�10, 2019.

�There is a significant need for novel treatment options for patients with MCL, especially those whose disease has progressed following several lines of previous therapy,� said Michael Wang, MD, ZUMA-2 Lead Investigator and Professor, Department of Lymphoma and Myeloma, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center. �The impressive response rates observed with KTE X19 support its potential as the first cell therapy for people living with MCL.�

With a median follow-up of 12.3 months (range: 7.0 to 32.3 months) at the time of data cutoff, 57 percent of patients remained in an ongoing response. Of the first 28 patients treated (minimum follow-up of 24 months), 43 percent were alive and remained in continued remission without additional therapy. The 12-month estimates of progression-free survival (PFS) and overall survival (OS) were 61 percent and 83 percent, respectively. Median duration of response, PFS and OS were not yet reached.

Among the 68 patients evaluable for safety, cytokine release syndrome (CRS) and neurologic events were observed in 91 percent and 63 percent of patients, respectively. Grade 3 or higher CRS and neurologic events were seen in 15 percent and 31 percent of patients, respectively. No Grade 5 CRS or neurologic events occurred.

Based on the results of the trial, Kite plans to submit a Biologics License Application (BLA) for KTE X19 to the FDA by the end of this year and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the first quarter of 2020. KTE X19 has been granted Breakthrough Therapy Designation (BTD) by the FDA and Priority Medicines (PRIME) by the EMA for relapsed or refractory MCL based on interim data from ZUMA-2.

About ZUMA-2 : ZUMA-2 is a single-arm, multicenter, open-label Phase II study involving 74 enrolled/leukapheresed adult patients ( greater than 18 years old) with MCL whose disease is refractory to or has relapsed following up to five prior lines of therapy, including anthracycline or bendamustine-containing chemotherapy, anti-CD20 monoclonal antibody therapy and the BTK inhibitors ibrutinib or acalabrutinib. The objectives of the study are to evaluate the efficacy (60 patients) and safety (68 patients) after a single infusion of KTE X19 in this patient population. The primary endpoint for the study is objective response rate (ORR). ORR in this trial is defined as the combined rate of complete responses and partial responses as assessed by an Independent Radiology Review Committee. Secondary endpoints include duration of response, best objective response, progression-free survival, overall survival, incidence of adverse events, incidence of anti-CD19 CAR antibodies, levels of anti-CD19 CAR T cells in blood, levels of cytokines in serum, and changes over time in the EQ-5D scale score and visual analogue scale score. The study is ongoing.